GC Pharma’s Enzyme Replacement Therapy Wins Orphan Drug Designation from EMA

After winning a nod from Japan at the start of 2021, GC Pharma’s Hunter syndrome treatment today received the orphan drug designation from EMA. The therapy, Hunterase ICV, is the world’s first enzyme replacement therapy for Hunter syndrome by ICV injection.

70% Hunter syndrome Patients Have CNS Symptoms

Hunter syndrome is a male-prevalent genetic disease, where the patient lacks the enzymes that can break down excessive lipids and sugars in the body; therefore, mucopolysaccharides build up in the body and affect the development of bone, organs, and nerves.

The possible symptoms that occur in the nerves, bones, joints, and organs are delayed growth at 5 years old, enlarged spleen and liver, hearing loss, a larger than normal head, and a short neck. Additionally, over 70% of individuals suffer damage to the central nervous system, while the average lifespan is around 10 to 20 years. Currently, there is no cure for the disease; patients could only rely on the standard of care, enzyme replacement therapy (ERT) with symptomatic therapy.

GC Pharma’s Hunterase ICV is able to deliver directly to the cerebral parenchyma, allowing it to reach the cells of the brain and central nervous system.  This sets it apart from the intravenous formulation, which is not able to pass the blood-brain barrier and reach the functional tissue in the human brain.

A 70% Drop in Heparan Sulfate

“This designation will be an important milestone toward addressing the urgent, unmet needs of families and patients living with Hunter syndrome,” said EC Huh, Ph.D., President of GC Pharma.

The designation is based on the positive Phase I/II trial of Hunterase ICV. In the trial, use of the drug is associated with a 70% decrease in heparan sulfate, a causative factor of delayed cognitive development in Hunter syndrome, in cerebrospinal fluid.

Earlier in January, Hunterase ICV was approved in Japan to treat delayed psychomotor development in patients with Hunter syndrome. With that, the drug became the first and only drug to treat central nervous system symptoms in patients with Hunter syndrome.

Author

Tyler Chen

"Never go to a doctor whose office plants have died."- Erma Bombeck